BR112019001887A2 - compositions and methods for treating cep290-associated disease - Google Patents

compositions and methods for treating cep290-associated disease

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Publication number
BR112019001887A2
BR112019001887A2 BR112019001887A BR112019001887A BR112019001887A2 BR 112019001887 A2 BR112019001887 A2 BR 112019001887A2 BR 112019001887 A BR112019001887 A BR 112019001887A BR 112019001887 A BR112019001887 A BR 112019001887A BR 112019001887 A2 BR112019001887 A2 BR 112019001887A2
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Brazil
Prior art keywords
cep290
associated disease
compositions
methods
treating
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BR112019001887A
Other languages
Portuguese (pt)
Inventor
Stefanidakis Michael
Lee Maeder Morgan
J Mepani Rina
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Editas Medicine Inc
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Application filed by Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of BR112019001887A2 publication Critical patent/BR112019001887A2/en

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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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Abstract

a presente invenção refere-se a ácidos nucleicos e vetores virais, particularmente vetores adeno-associados a vírus (aav) que codificam cas9 e rnas guia emparelhados. os ácidos nucleicos e vetores, e composições que os compreendem, podem ser usados em métodos para tratar indivíduos, para alterar células em indivíduos que podem sofrer de uma distrofia retinal herdada tal como doença associada a cep290 ou que podem estar precisando de alteração de uma célula ou de uma sequência de ácido nucleico celular associada com uma distrofia retinal herdada tal como cep290 genética, e/ou para tratar distrofias retinais herdadas incluindo doença associada a cep290.The present invention relates to nucleic acids and viral vectors, particularly adeno-associated virus (aav) vectors encoding paired guide and rna. nucleic acids and vectors, and compositions comprising them, may be used in methods for treating individuals, for altering cells in individuals who may suffer from an inherited retinal dystrophy such as cep290-associated disease or may need to change a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as genetic cep290, and / or to treat inherited retinal dystrophies including cep290-associated disease.

BR112019001887A 2016-08-02 2017-08-02 compositions and methods for treating cep290-associated disease BR112019001887A2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201662370202P 2016-08-02 2016-08-02
US201662400526P 2016-09-27 2016-09-27
US201762443568P 2017-01-06 2017-01-06
US201762503800P 2017-05-09 2017-05-09
US201762535193P 2017-07-20 2017-07-20
PCT/US2017/045191 WO2018026976A1 (en) 2016-08-02 2017-08-02 Compositions and methods for treating cep290 associated disease

Publications (1)

Publication Number Publication Date
BR112019001887A2 true BR112019001887A2 (en) 2019-07-09

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BR112019001887A BR112019001887A2 (en) 2016-08-02 2017-08-02 compositions and methods for treating cep290-associated disease

Country Status (6)

Country Link
US (2) US11566263B2 (en)
EP (1) EP3494220A1 (en)
AU (2) AU2017305404B2 (en)
BR (1) BR112019001887A2 (en)
CA (1) CA3032822A1 (en)
WO (1) WO2018026976A1 (en)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (en) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 AAV delivery of nucleobase editor
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3601562A1 (en) 2017-03-23 2020-02-05 President and Fellows of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
EP3676376A2 (en) 2017-08-30 2020-07-08 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20200121782A (en) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 Uses of adenosine base editor
AU2019237541A1 (en) 2018-03-23 2020-10-08 Editas Medicine, Inc. CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated Usher syndrome
US11981892B2 (en) 2018-04-16 2024-05-14 University Of Massachusetts Compositions and methods for improved gene editing
AU2019314208A1 (en) * 2018-08-02 2021-02-18 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
BR112021018606A2 (en) 2019-03-19 2021-11-23 Harvard College Methods and compositions for editing nucleotide sequences
CA3169991A1 (en) * 2020-02-12 2021-08-19 Massachusetts Eye And Ear Infirmary Haplotype-based treatment of rp1 associated retinal degenerations
CN111471712A (en) * 2020-04-22 2020-07-31 江苏同科医药科技有限公司 Construction method of CRISPR/Cas9 double-target-knockout target gene vector
DE112021002672T5 (en) 2020-05-08 2023-04-13 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EDIT BOTH STRANDS SIMULTANEOUSLY OF A DOUBLE STRANDED NUCLEOTIDE TARGET SEQUENCE
WO2024026478A1 (en) * 2022-07-29 2024-02-01 Beam Therapeutics Inc. Compositions and methods for treating a congenital eye disease

Family Cites Families (109)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
US5869624A (en) 1993-03-26 1999-02-09 Progenics Pharmaceuticals, Inc. HIV-1 vaccines, antibody compositions related thereto, and therapeutic and prophylactic uses thereof
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
US5725493A (en) 1994-12-12 1998-03-10 Avery; Robert Logan Intravitreal medicine delivery
US6299895B1 (en) 1997-03-24 2001-10-09 Neurotech S.A. Device and method for treating ophthalmic diseases
US6203986B1 (en) 1998-10-22 2001-03-20 Robert H. Singer Visualization of RNA in living cells
EP1154691A4 (en) 1999-01-05 2004-07-07 Massachusetts Eye & Ear Infirm Targeted transscleral controlled release drug delivery to the retina and choroid
ES2231257T3 (en) 1999-10-21 2005-05-16 Alcon Inc. DEVICE FOR THE ADMINISTRATION OF PHARMACOS.
US6416777B1 (en) 1999-10-21 2002-07-09 Alcon Universal Ltd. Ophthalmic drug delivery device
KR100381902B1 (en) 1999-11-29 2003-04-26 아이진 주식회사 A glaucoma-coding DNA and protein
JP2004536631A (en) 2001-05-03 2004-12-09 マサチューセッツ・アイ・アンド・イア・インファーマリー Implantable drug delivery device and use thereof
AU2003219847A1 (en) 2002-02-21 2003-09-09 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
EP1532178A4 (en) 2002-06-11 2006-10-25 Scripps Research Inst Artificial transcription factors
AU2005287365B2 (en) 2004-08-23 2011-02-10 Alnylam Pharmaceuticals, Inc. Multiple RNA polymerase III promoter expression constructs
WO2007014181A2 (en) 2005-07-25 2007-02-01 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007106690A2 (en) 2006-03-15 2007-09-20 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
BRPI0808704B1 (en) 2007-03-02 2022-01-18 Dupont Nutrition Biosciences Aps METHOD TO GENERATE AN INITIAL CULTURE COMPRISING AT LEAST TWO BACTERIOPHAGE-RESISTANT VARIANTS, INITIATOR CULTURE AND FERMENTATION METHOD
US20110117058A1 (en) * 2008-04-02 2011-05-19 Fondazione Telethon Method of treating genetic disorders
US8546553B2 (en) 2008-07-25 2013-10-01 University Of Georgia Research Foundation, Inc. Prokaryotic RNAi-like system and methods of use
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
US9115386B2 (en) 2008-09-26 2015-08-25 Children's Medical Center Corporation Selective oxidation of 5-methylcytosine by TET-family proteins
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
BR112012028805A2 (en) 2010-05-10 2019-09-24 The Regents Of The Univ Of California E Nereus Pharmaceuticals Inc endoribonuclease compositions and methods of use thereof.
CN103025344B (en) 2010-05-17 2016-06-29 桑格摩生物科学股份有限公司 Novel DNA-associated proteins and application thereof
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
GB201013153D0 (en) 2010-08-04 2010-09-22 Touchlight Genetics Ltd Primer for production of closed linear DNA
US9499592B2 (en) 2011-01-26 2016-11-22 President And Fellows Of Harvard College Transcription activator-like effectors
EP3254703B1 (en) 2011-04-22 2020-02-19 The Regents of The University of California Adeno-associated virus virions with variant capsid and methods of use thereof
US20140113376A1 (en) 2011-06-01 2014-04-24 Rotem Sorek Compositions and methods for downregulating prokaryotic genes
EP3378938A1 (en) 2011-06-10 2018-09-26 Institut National de la Sante et de la Recherche Medicale (INSERM) Methods for the treatment of leber congenital amaurosis
EP2732038B1 (en) 2011-07-15 2018-09-05 The General Hospital Corporation Methods of transcription activator like effector assembly
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
EP2820159B1 (en) 2012-02-29 2019-10-23 Sangamo Therapeutics, Inc. Methods and compositions for treating huntington's disease
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
ES2960803T3 (en) 2012-05-25 2024-03-06 Univ California Methods and compositions for RNA-directed modification of target DNA and for modulation of RNA-directed transcription
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
CN116622704A (en) 2012-07-25 2023-08-22 布罗德研究所有限公司 Inducible DNA binding proteins and genomic disruption tools and uses thereof
EP2880171B1 (en) 2012-08-03 2018-10-03 The Regents of The University of California Methods and compositions for controlling gene expression by rna processing
KR102474010B1 (en) 2012-08-29 2022-12-02 상가모 테라퓨틱스, 인코포레이티드 Methods and compositions for treatment of a genetic condition
EP2906602B1 (en) 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
ES2926021T3 (en) 2012-10-23 2022-10-21 Toolgen Inc Composition for cleaving a target DNA comprising a target DNA-specific guide RNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof
EP3617309A3 (en) 2012-12-06 2020-05-06 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG10201912328UA (en) 2012-12-12 2020-02-27 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
EP3144390B1 (en) 2012-12-12 2020-03-18 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
CN113355357A (en) 2012-12-12 2021-09-07 布罗德研究所有限公司 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
ES2576128T3 (en) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modification by genetic technology and optimization of systems, methods and compositions for the manipulation of sequences with functional domains
CN114634950A (en) 2012-12-12 2022-06-17 布罗德研究所有限公司 CRISPR-CAS component systems, methods, and compositions for sequence manipulation
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
IL239344B1 (en) 2012-12-12 2024-02-01 Broad Inst Inc Engineering of systems, methods and optimized guide compositions for sequence manipulation
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
EP3623463B1 (en) 2013-02-07 2021-10-20 The General Hospital Corporation Tale transcriptional activators
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
WO2015099850A1 (en) 2013-12-26 2015-07-02 The General Hospital Corporation Multiplex guide rnas
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US10119133B2 (en) 2013-03-15 2018-11-06 The General Hospital Corporation Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
EP3730615A3 (en) 2013-05-15 2020-12-09 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
EP3603679B1 (en) 2013-06-04 2022-08-10 President and Fellows of Harvard College Rna-guided transcriptional regulation
CA2914519A1 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
AU2014281028B2 (en) 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
JP2016528890A (en) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ Therapeutic use of genome editing using the CRISPR / Cas system
RU2764637C2 (en) 2013-07-09 2022-01-19 Президент Энд Фэллоуз Оф Харвард Коллидж Multiplex genomic engineering guided by rna
SG10201913015XA (en) 2013-07-10 2020-02-27 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
WO2015020522A1 (en) 2013-08-05 2015-02-12 Koninklijke Nederlandse Akademie Van Wetenschappen Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015070083A1 (en) 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS
JP2016537028A (en) 2013-11-18 2016-12-01 クリスパー セラピューティクス アーゲー CRISPR-CAS System Materials and Methods
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
CN111206032A (en) 2013-12-12 2020-05-29 布罗德研究所有限公司 Delivery, use and therapeutic applications of CRISPR-CAS systems and compositions for genome editing
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
EP3114227B1 (en) * 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US9970001B2 (en) 2014-06-05 2018-05-15 Sangamo Therapeutics, Inc. Methods and compositions for nuclease design
JP6930834B2 (en) 2014-06-16 2021-09-01 ザ・ジョンズ・ホプキンス・ユニバーシティー Compositions and Methods for Expression of CRISPR Guide RNA Using the H1 Promoter
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
EP3274453B1 (en) 2015-03-26 2021-01-27 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
US20160324987A1 (en) 2015-04-15 2016-11-10 Cedars-Sinai Medical Center Use of crispr/cas9 as in vivo gene therapy to generate targeted genomic disruptions in genes bearing dominant mutations for retinitis pigmentosa
JP7030522B2 (en) 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド Optimized CRISPR / CAS9 system and method for gene editing in stem cells
BR112017024514A2 (en) * 2015-05-16 2018-07-24 Genzyme Corporation genetic editing of deep intronic mutations
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
ES2929110T3 (en) 2015-08-25 2022-11-24 Univ Duke Compositions and methods to improve the specificity in genetic engineering using RNA-guided endonucleases
WO2017040511A1 (en) 2015-08-31 2017-03-09 Agilent Technologies, Inc. Compounds and methods for crispr/cas-based genome editing by homologous recombination
WO2017184768A1 (en) 2016-04-19 2017-10-26 The Broad Institute Inc. Novel crispr enzymes and systems
KR20190039703A (en) 2016-07-05 2019-04-15 더 존스 홉킨스 유니버시티 CRISPR / CAS9-based compositions and methods for treating retinal degeneration
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
CN110249052B (en) 2016-12-30 2024-04-12 爱迪塔斯医药公司 Synthetic guide molecules, compositions, and methods related thereto
EP4095263A1 (en) 2017-01-06 2022-11-30 Editas Medicine, Inc. Methods of assessing nuclease cleavage

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